ATLAS-INH: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients with Hemophilia A or B, with Inhibitory Antibodies to Factor VIII or IX

ATLAS-A/B: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients with Hemophilia A or B, without Inhibitory Antibodies to Factor VIII or IX

The Division of Kidney, Urologic, and Hematologic Diseases (DKUHD) of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), in collaboration with the National Institute of Child Health and Human Development (NICHD)

LCH IV International Collaborative Treatment Protocol for Children and Adolescents with Langerhans Cell Histiocytosis

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Investigate the Efficacy and Safety of Mongersen (GED-0301) for the Treatment of Adult and Adolescent Subjects with Active Crohn’s Disease

Phase 3b, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess the Safety, Efficacy, and Tolerability of Tezacaftor/Ivacaftor (TEZ/IVA) in an Orkambi-experienced Population Who Are Homozygous for the F508del CFTR Mutation

A Multi-center Study to Evaluate the Pharmacokinetics of Diacerein and Rhein and the Safety of Diacerein after Maximum Use, Topical Administration of CCP-020 (Diacerein 1% ointment) to Patients with Epidermolysis Bullosa (EB)

Functional Outcomes following Musculoskeletal Coccidioidomycosis: Mid-Term Results at 2 Years

Parent-Child Disclosures About Chronic Conditions

The impact of pediatric skin disorders: The big study

The role of executive function on health-related quality of life in children and adolescents with congenital or acquired heart disease who have undergone cardiothoracic surgery